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PepGen To Voluntarily Pause Mid-Stage Duchenne Muscular Dystrophy Study, Stock Falls
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PepGen To Voluntarily Pause Mid-Stage Duchenne Muscular Dystrophy Study, Stock Falls
Mar 4, 2025 10:59 AM

On Tuesday, PepGen Inc. ( PEPG ) announced its voluntary decision to temporarily pause the Phase 2 CONNECT2-EDO51 study of PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD) until the company can review results from the 10 mg/kg cohort.

The first two cohorts of the CONNECT1 study are fully enrolled and data from the 10 mg/kg cohort are expected during the third quarter of 2025. No new safety issues related to PGN-EDO51 have been observed since the company’s last safety update as of January 23, 2025.

Also Read: Capricor Therapeutics Cell Therapy Goes Under Priority FDA Review For Duchenne Associated-Heart Condition

“With our 10 mg/kg cohort of CONNECT1 study fully enrolled and data expected later this year, we decided to pause CONNECT2 until we are able to review results from the 10 mg/kg cohort in patients with DMD. This will allow us to gather additional safety data, assess the impact of this dose of PGN-EDO51 on dystrophin levels, and potentially improve the design of CONNECT2,” said James McArthur, president and CEO of PepGen ( PEPG ).

“This decision enables us to focus resources on completing CONNECT1, as well as rapidly advancing our FREEDOM studies in myotonic dystrophy type 1 with PGN-EDODM1, in which we recently reported encouraging initial clinical data from the Phase 1 FREEDOM-DM1 study,” McArthur added.

In February, PepGen ( PEPG ) announced early data from its FREEDOM-DM1 trial for myotonic dystrophy.

The study showed significant dose-dependent splicing correction, with mean corrections of 12.3% at 5 mg/kg and 29.1% at 10 mg/kg at 28 days post-dosing.

Price Action: PEPG stock is down 21% at $2.22 at the last check Tuesday.

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